NHS to Fast Track as Yet Unapproved Cholesterol Drug 

Nicky Broyd

January 14, 2020

The NHS in England has agreed early access to a new, as yet unapproved, cholesterol drug in what it described as a world-first 'population-level agreement'. In a collaboration with the manufacturer and the University of Oxford the drug, Inclisiran (Novartis), will also be studied as part of a large-scale NHS clinical trial.

Inclisiran is predicted to save around 30,000 lives over the next 10 years, the Department for Health and Social Care (DHSC) said.

The drug is injected twice a year which could improve adherence to treatment.

It prevents production of the PCSK9 molecule to lower LDL blood cholesterol and is said to have few side effects.

Accelerated Approval

No financial details of the deal have been made public but there's a commitment for it to be accelerated through the National Institute for Health and Care Excellence’s (NICE) funding approval programme. NHS clinical trial data will also be collected.

The drug is expected to be submitted for European Medicines Agency approval in the near future and could be available on the NHS as early as next year. It has not yet been approved by the US FDA either.

In a statement, the Chair of NHS England, Lord Prior, said: "This innovative and groundbreaking collaboration could transform the health outlook of tens of thousands of people suffering from heart disease, by bringing together in a unique combination our ability to organise large scale clinical trials, to address highly complex manufacturing issues, and to reach a large population of patients.

"It is a great illustration of how the UK Life Sciences Strategy can help both NHS patients and the wider economy, and shows that the UK can be the centre of a dynamic life sciences ecosystem whilst delivering great care."

Professor of Medicine and Epidemiology at the University of Oxford Martin Landray said: "This trial provides an opportunity to demonstrate how a highly streamlined trial can be conducted within the UK by combining elements of patients already diagnosed cardiovascular disease and received treatment through the successful ORION 4 trial with the high-throughput clinics developed for UK Biobank.

"The trial will provide both a very reliable test of the efficacy and safety of inclisiran to support a population-health approach to the management of cholesterol, and act as an exemplar for future trials of other treatments in the UK."

Novartis acquired the drug when it bought The Medicines Company earlier this month.

Novartis CEO Vas Narasimhan said it was "a unique opportunity with Inclisiran to use innovative population based approaches to open up a new chapter for the treatment of cardiovascular disease, the world’s leading cause of mortality and disability."

He added that the partnership would "leverage innovative models that could potentially lead to generating leading scientific evidence, accelerate access for patients and ensure continuous improvement in manufacturing efficiency and optimisation".

'Exciting Announcement'

Experts have been commenting on the deal through the Science Media Centre.

Dr Riyaz Patel, associate professor and consultant cardiologist, Barts Health NHS Trust, said: "This is a really exciting announcement that changes the way we bring new medicines to patients earlier and also propels the NHS and the UK as a world leader in this sort of clinical research."

However, he questioned the life-saving prediction made by DHSC: "Whether it will save 30,000 lives depends on many factors. It is an estimate based on extrapolation from existing data with multiple assumptions. The trials for example included only specific patients (high risk or with known heart disease), and typically not everyone may be eligible to get the drug as there will be exclusions."

That theme was picked up by Prof Stephen MacMahon, principal director, The George Institute for Global Health: "Before concluding that 30,000 lives might be saved in the UK, it might be prudent to await the results of the trial and not prejudge its outcome."

Prof Kausik Ray, professor of public health, Imperial College London, said: "Inclisiran is an investigational drug which is unapproved at present but could be here as early as 2021. The ORION 1 and 11 trials which I led showed the potential of twice yearly dosing to reduce LDL cholesterol by 50%. The potential of this therapy is enormous from a convenience point of view and has the potential to overcome a huge issue which is non-adherence to medications which need to be taken daily. So far, the data suggests the drug is safe and the ongoing ORION 4 trial is testing the possibility of this approach. This approach promises to revolutionise cardiovascular disease prevention."

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